AU2019252529A1 - Double-stranded nucleic acid inhibitor molecules modified with Tm-increasing nucleotides - Google Patents
Double-stranded nucleic acid inhibitor molecules modified with Tm-increasing nucleotides Download PDFInfo
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- AU2019252529A1 AU2019252529A1 AU2019252529A AU2019252529A AU2019252529A1 AU 2019252529 A1 AU2019252529 A1 AU 2019252529A1 AU 2019252529 A AU2019252529 A AU 2019252529A AU 2019252529 A AU2019252529 A AU 2019252529A AU 2019252529 A1 AU2019252529 A1 AU 2019252529A1
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- nucleic acid
- double
- acid inhibitor
- nucleotides
- nucleotide
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- C12Y—ENZYMES
- C12Y304/00—Hydrolases acting on peptide bonds, i.e. peptidases (3.4)
- C12Y304/21—Serine endopeptidases (3.4.21)
- C12Y304/21061—Kexin (3.4.21.61), i.e. proprotein convertase subtilisin/kexin type 9
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- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/14—Type of nucleic acid interfering nucleic acids [NA]
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/322—2'-R Modification
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/323—Chemical structure of the sugar modified ring structure
- C12N2310/3231—Chemical structure of the sugar modified ring structure having an additional ring, e.g. LNA, ENA
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/35—Nature of the modification
- C12N2310/351—Conjugate
- C12N2310/3519—Fusion with another nucleic acid
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/52—Physical structure branched
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/531—Stem-loop; Hairpin
-
- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/50—Physical structure
- C12N2310/53—Physical structure partially self-complementary or closed
- C12N2310/533—Physical structure partially self-complementary or closed having a mismatch or nick in at least one of the strands
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/50—Methods for regulating/modulating their activity
- C12N2320/52—Methods for regulating/modulating their activity modulating the physical stability, e.g. GC-content
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- Health & Medical Sciences (AREA)
- Life Sciences & Earth Sciences (AREA)
- Engineering & Computer Science (AREA)
- Genetics & Genomics (AREA)
- Biomedical Technology (AREA)
- Chemical & Material Sciences (AREA)
- Organic Chemistry (AREA)
- General Engineering & Computer Science (AREA)
- Wood Science & Technology (AREA)
- Zoology (AREA)
- Bioinformatics & Cheminformatics (AREA)
- Molecular Biology (AREA)
- Biotechnology (AREA)
- General Health & Medical Sciences (AREA)
- Biochemistry (AREA)
- Physics & Mathematics (AREA)
- Biophysics (AREA)
- Plant Pathology (AREA)
- Microbiology (AREA)
- Medicinal Chemistry (AREA)
- Pharmacology & Pharmacy (AREA)
- Epidemiology (AREA)
- Animal Behavior & Ethology (AREA)
- Public Health (AREA)
- Veterinary Medicine (AREA)
- Virology (AREA)
- Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
- Medicinal Preparation (AREA)
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
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| US201862657428P | 2018-04-13 | 2018-04-13 | |
| US62/657,428 | 2018-04-13 | ||
| US201862778755P | 2018-12-12 | 2018-12-12 | |
| US62/778,755 | 2018-12-12 | ||
| PCT/US2019/027021 WO2019200124A1 (en) | 2018-04-13 | 2019-04-11 | Double-stranded nucleic acid inhibitor molecules modified with tm-increasing nucleotides |
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| AU2019252529A1 true AU2019252529A1 (en) | 2020-10-01 |
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| AU2019252529A Abandoned AU2019252529A1 (en) | 2018-04-13 | 2019-04-11 | Double-stranded nucleic acid inhibitor molecules modified with Tm-increasing nucleotides |
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| US (1) | US11873488B2 (https=) |
| EP (1) | EP3773609A4 (https=) |
| JP (1) | JP2021520355A (https=) |
| KR (1) | KR20200144100A (https=) |
| CN (1) | CN111971051A (https=) |
| AU (1) | AU2019252529A1 (https=) |
| CA (1) | CA3094008A1 (https=) |
| IL (1) | IL277813A (https=) |
| MX (1) | MX2020010802A (https=) |
| WO (1) | WO2019200124A1 (https=) |
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| AU2019397247A1 (en) * | 2018-12-12 | 2021-06-24 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules containing a triloop |
| WO2020226960A1 (en) * | 2019-05-03 | 2020-11-12 | Dicerna Pharmaceuticals, Inc. | Double-stranded nucleic acid inhibitor molecules with shortened sense strands |
| US20240287512A1 (en) * | 2021-05-11 | 2024-08-29 | Dicerna Pharmaceuticals, Inc. | Lipid conjugation for targeting neurons of the central nervous system |
| WO2023069707A2 (en) * | 2021-10-22 | 2023-04-27 | Sirnaomics, Inc. | Products and compositions |
| JPWO2023176863A1 (https=) * | 2022-03-16 | 2023-09-21 |
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| US8273866B2 (en) * | 2002-02-20 | 2012-09-25 | Merck Sharp & Dohme Corp. | RNA interference mediated inhibition of gene expression using chemically modified short interfering nucleic acid (SINA) |
| WO2009117589A1 (en) | 2008-03-21 | 2009-09-24 | Isis Pharmaceuticals, Inc. | Oligomeric compounds comprising tricyclic nucleosides and methods for their use |
| JP2012504389A (ja) * | 2008-09-22 | 2012-02-23 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | 修飾を有するｄｓＲＮＡによる遺伝子発現の特異的な阻害のための組成物および方法 |
| US20110288147A1 (en) | 2008-09-22 | 2011-11-24 | Bob Dale Brown | Compositions and methods for the specific inhibition of gene expression by DSRNA containing a tetraloop |
| AU2009336191B2 (en) * | 2008-12-18 | 2017-08-24 | Novo Nordisk A/S | Extended dicer substrate agents and methods for the specific inhibition of gene expression |
| CN104651362A (zh) * | 2009-04-03 | 2015-05-27 | 戴瑟纳制药公司 | 利用不对称双链rna特异性抑制kras的方法和组合物 |
| US20140011860A1 (en) | 2010-07-19 | 2014-01-09 | Isis Pharmaceuticals, Inc. | Compounds and methods for modulating target nuclear and sub-nuclear nucleic acid molecules in cells and animals |
| EP2850092B1 (en) | 2012-04-09 | 2017-03-01 | Ionis Pharmaceuticals, Inc. | Tricyclic nucleic acid analogs |
| CA2902571A1 (en) * | 2013-03-15 | 2014-09-18 | MiRagen Therapeutics, Inc. | Bridged bicyclic nucleosides |
| WO2014140348A1 (en) | 2013-03-15 | 2014-09-18 | Universität Bern | Tricyclic nucleosides and oligomeric compounds prepared therefrom |
| DK3444350T3 (da) * | 2013-07-03 | 2022-02-07 | Dicerna Pharmaceuticals Inc | Fremgangsmåder og sammensætninger til specifik hæmning af alpha-1 antitrypsin ved dobbeltstrenget rna |
| US10150965B2 (en) * | 2013-12-06 | 2018-12-11 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of transthyretin (TTR) by double-stranded RNA |
| EP3119789B1 (en) | 2014-03-17 | 2020-04-22 | Ionis Pharmaceuticals, Inc. | Bicyclic carbocyclic nucleosides and oligomeric compounds prepared therefrom |
| AU2015330670B2 (en) * | 2014-10-10 | 2022-01-06 | Novo Nordisk Health Care Ag | Therapeutic inhibition of lactate dehydrogenase and agents therefor |
| JP7105065B2 (ja) * | 2014-12-15 | 2022-07-22 | ダイセルナ ファーマシューティカルズ， インコーポレイテッド | リガンド修飾二本鎖核酸 |
| US10036017B2 (en) * | 2015-02-17 | 2018-07-31 | Dicerna Pharmaceuticals, Inc. | Methods and compositions for the specific inhibition of complement component 5(C5) by double-stranded RNA |
| KR102350647B1 (ko) | 2016-09-02 | 2022-01-14 | 다이서나 파마수이티컬, 인크. | 4'-포스페이트 유사체 및 이를 포함하는 올리고뉴클레오타이드 |
| KR20200023427A (ko) * | 2017-06-29 | 2020-03-04 | 다이서나 파마수이티컬, 인크. | Hmgb1 발현을 억제하기 위한 조성물 및 방법 |
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- 2019-04-11 JP JP2020554092A patent/JP2021520355A/ja not_active Withdrawn
- 2019-04-11 EP EP19784831.0A patent/EP3773609A4/en active Pending
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2020
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Also Published As
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| JP2021520355A (ja) | 2021-08-19 |
| MX2020010802A (es) | 2020-10-28 |
| EP3773609A4 (en) | 2022-05-18 |
| KR20200144100A (ko) | 2020-12-28 |
| IL277813A (en) | 2020-11-30 |
| US20190316125A1 (en) | 2019-10-17 |
| WO2019200124A1 (en) | 2019-10-17 |
| US11873488B2 (en) | 2024-01-16 |
| EP3773609A1 (en) | 2021-02-17 |
| CN111971051A (zh) | 2020-11-20 |
| CA3094008A1 (en) | 2019-10-17 |
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